Sobi Capital Markets Day
2020
10 December 2020
Paula Treutiger
Head of Communications and Investor RelationsWelcome
2
In order to utilise the ‘Safe Harbor’ provisions of the United States Private Securities Litigation Reform Act of 1995, Swedish Orphan Biovitrum AB (publ) is providing the following cautionary statement. This presentation contains forward-looking statements with respect to the financial condition, results of operations and businesses of Swedish Orphan Biovitrum AB (publ), By their nature, forward-looking statements and forecasts involve risk and uncertainty because they relate to events and depend on circumstances that will occur in the future. There are a number of factors that could cause actual results and developments to differ materially from that expressed or implied by these forward-looking statements. These factors include, among other things, the loss or expiration of patents, marketing exclusivity or trade marks; exchange rate fluctuations; the risk that R&D will not yield new products that achieve commercial success; the impact of competition, price controls and price reductions; taxation risks; the risk of substantial product liability claims; the impact of any failure by third parties to supply materials or services; the risk of delay to new product launches; the difficulties of obtaining and maintaining governmental approvals for products; the risk of failure to observe ongoing regulatory oversight; the risk that new products do not perform as we expect; and the risk of environmental liabilities.
Forward-looking statements
3
Guido OelkersCEO
Ravi RaoHead of R&D and CMO
Norbert OppitzHead of Immunology and International
Henrik StenqvistCFO
4
Today’s presenters
Today’s agenda
5
14:45Internationalisation strategy Norbert Oppitz
15:15Wrap up and Q&A Guido Oelkers
Financial update 15:00Henrik Stenqvist
13:05Strategy and realising opportunities Guido Oelkers
13:30 Innovation management at Sobi Ravi Rao
Break 14:30
Guido Oelkers
CEO
Strategy and realisingopportunities
6
Five years ago, Sobi looked very different
Revenue 2016
7
48% Haemophilia52%All other
Significant dependence on an emerging haemophilia portfolio
Pipeline with no big bets in rather early-stage R&D
Europe-centric approach without significant footprint in the US
Business largely characterised by in-licensing/ distributor agreements
International business sub-scale in most geographies outside Europe
SEK 5.2B
Source: Sobi
SEK B
~75% of growth is organic1
EBITA %
We have accomplished much in the past four years
52016
25%75%2020E 15-15.5
x2.7 1.5
5.7-6.2
x3.8
Revenue EBITA
30%
39%
Source: Sobi Annual Report 2016; 2020 guidance per Q3 2020 report 8
Organic growth Inorganic growth
1. Inorganic growth defined as FY 2018 revenue for Synagis (USD 287M) and Doptelet (USD 10M)
Committed to a continuous upgrade of our sustainability agenda
Sustainability strategy
• We have no tolerance for corruption
• We are transparent
• We source responsibly
• We develop our people and keep them safe and healthy
• We reduce our environmental footprint
• Our R&D is ethical and focused on medical need
• We expand access to treatment
• We are patientcentric & engage with our communities
• We contribute to knowledge to enhance the practice of medicine
• We focus on patient safety
Commitment to patients Responsible behavior
Commitment to Agenda 2030 and the Paris Agreement
9
1. Drive
Haemophiliapenetration
4. Strengthen
late-stage pipeline
3. Grow US business
and strengthenposition inEMENAR
2. Develop
Immunology
In 2017, we set out our strategy to build a regional leader in rare disease – a strategy on which we have forcefully delivered
Source: Sobi Annual Report 2017 10
1. We have made a significant impact in the haemophilia market
22%Prophylaxis
patient market share1
35%Prophylaxis
patient market share2
#1Ranked leader in haemophilia by healthcare professionals3
From
2023we advance our
leadership through BIVV001 as new
normal
11
#1in community and scientific engagement4
BIVV001 is developed and, if approved, will be commercialised in collaboration with Sanofi
1. Elocta FY sales for 2019 in relation to estimates based on MRB, Global Forecast of the Factor VIII Market by Region, Product Category and Company to 2022, November 2018; Data for Europe and Middle East and Africa 2. Internal data 3. Corporate Perception survey with HCPs treating haemophilia 2016 – 2019, N= 48 4. Internal data
2. Immunology: strong performance since establishment in 2016
Immunology revenue share Driven by three major products
81%
19%
2016
2020E65%
35%
Immunology Other
Performing well under our ownership
Growing strongly at 27% YTD
Established biology leading us into new fields
H1 2019
H1 2018
H2 2018
1,052
H2 2019
1,008
1,781
1,464
H1 2020
1,248 +19%
Acquisition Transition complete
Revenue, SEK M
1,493
Jan-Sep2019
1,175
Jan-Sep2020
+27%
Revenue, SEK M
First targeted treatment forprimary haemophagocytic lymphohistiocytosis (pHLH), a life-threatening hyper-inflammation disease
12Source: Sobi
3. US business now a powerful platform for future growth
0.9
4.3
US Non-US
Over the past five years, we have increased our footprint in the US to ~35% of total sales
Key drivers for increasing importance of US business were acquisitions of Synagis, Gamifant and Doptelet
Going forward, US will continue to grow both in absolute and relative terms due to Doptelet and Gamifant
Revenue by geography, SEK B
2016 2020E
5.3-5.5
9.7-10.0
82%18% ~65%~35%
Background
13Source: Sobi (2020E based on mid-point guidance provided by Sobi in Q3 2020 report)
4. Capturing substantial value from our late-stage pipeline1
1. Not all programmes have been started 2. In collaboration with Apellis 3. Strategic licensing agreement with Selecta 4. Financial interest only, in collaboration with AstraZeneca 5. BIVV001 is developed and, if approved, will be commercialised in collaboration with Sanofi 14
Phase 2 Phase 3 In registration
pegcetacoplan2
IC-MPGN and C3G
Gamifant / emapalumab
GvHD
Kineret / anakinra
Deficiency of IL-1 receptor antagonist (DIRA) (US)
SEL-212 / pegadricase3
Chronic refractory gout
pegcetacoplan2
ALS
pegcetacoplan2
CAD
Gamifant / emapalumab
Graft failure (GF)
MEDI8897 / nirsevimab4
RSV prevention
Doptelet / avatrombopag
Chronic immune thrombocytopenia (ITP) (EU)
pegcetacoplan 2nd line2
Paroxysmal nocturnal haemoglobinuria and other
BIVV001 / rFVIIIFc-VWF-XTEN25
Haemophilia A
pegcetacoplan2
HSCT-TMA
Gamifant / emapalumab
Secondary HLH rheumatology
Gamifant / emapalumab
Secondary HLH malignancy
Gamifant / emapalumab
Primary HLH (RoW)
pegcetacoplan 1st line2
Paroxysmal nocturnal haemoglobinuria and other
M&A continues to be a key driver for our transformation
20202018 2019
NovimmuneGamifant (NI-1701/NI-1801)
DovaDoptelet
AstraZenecaSynagis
nirsevimab/ MEDI8897
SelectaSEL-212
ApellisSystemic
pegcetacoplan
SanofiBIVV001 (opt-in)
BIVV001 is developed and, if approved, will be commercialised in collaboration with SanofiNirsevimab is being developed by AZ and commercialised by Sanofi. Sobi has an option to the rights to 50% of the profits and losses of the product in the US
15
How we will grow beyond today’s core
Existing (EU+US) New (International)
Geography
New
Current
Portfolio
2 Deliver on our late-stage pipeline and launch(60-70% of growth)
• 12 late-stage programs
• Develop Gamifant beyond pHLH
• Make Doptelet TPO-RA of choice
• Long-term leadership with BIVV001
Grow the core(5-10% of growth)
• Maintain leadership in haemophilia
• Expand Kineret into CSS
Go global(20-30% of growth)
• China
• Japan
• Russia
• Australia, South Korea
• Middle East
• CEE
• Latin America1
3
16
For the coming five years, our new ambition is ‘25 by 25’
516
6
3
20252016 1817 19 20
15-15.5
25±2
10
10
Grow the core3Capture value of pipeline2Go global1Revenuein SEK B
17
1. Growth from core and pipeline in international markets2. Growth from pipeline in current markets3. Growth from core in current markets
1. Committed to develop Elocta in a challenging environment
132
0
1,000
2,000
3,000
4,000
YTD product sales1
SEK M
YTD Q3
2,316
YTD Q3 YTD Q3 YTD Q3 YTD Q3
1,018
3,2743,514
• New launches, such as Russia, opportunity of SEK 500 million
• Penetration upside in certain markets
• Large patient pool on SHL and plasma
• Clear long-term commitment to patients with BIVV001 and normalisation
• Increasing competition from new EHLs and NFT
• Price pressure as a result of increasing competition and post COVID-19 cost constraints
• Access restrictions and reduction in per-capita consumption – COVID-19 prohibiting patients and pharma from visiting physicians and reducing surgical procedures
18
1. Excluding royalties2016 2017 2018 2019 2020
Source: Sobi
Opportunities Headwinds
1. Creating material growth from a strong trajectory and clear differentiation
2019 2020E
1.2
1.4
+19%
Doptelet is a small molecule thrombopoietin receptor agonist used to treat thrombo-cytopenia in ITP2 and CLD3
As a cost-effective oraltreatment with high efficacy and administered with food and without dietary restrictions, it is clearly differentiated against competitors and well on the way to becoming the TPO-RA of choice
We enable broader access to Doptelet through expansion into new markets, including in Europe and through our international divisionincluding Japan2,3
US TPO-RA1 market USD B
Source: Symphony Monthly Data (Sep 2020), RFT 11.4.2019
Sobi's market share in the USAdult ITP only
Sept 2019 Sept 2020
1.4%
6.3%
+350%
1. Thrombopoietin receptor agonist 2. Approved for ITP in the US and awaiting approval in the EU. See prescribing information in the applicable territory for the approved indication.3. Approved for patients with CLD and thrombocytopenia undergoing a procedure in US, Europe and China. See prescribing information in the applicable territory for the approved indication.
See prescribing information in the applicable territory for the approved indication.
2. Developing Gamifant: a strong trajectoryinto the future
YTD Q3 2019
78
YTD Q3 2020
126
+61%
Development of Gamifant over time, in thousands of mg
HLH
Maximise in pHLH
Expand geographically
Drive disease awareness
Expand into sHLH
Develop sHLH from rHLH and mHLH to iHLH
Move to the next frontier: GF & GvHD
Develop GF with companion diagnostic
HSCT
20Source: Sobi
21
2. We opted in early1 for BIVV001 – to drive our leadership in haemophilia into the future
21
Builds on the Fc-fusion technology – same as for Elocta – with added domains of von Willebrand factor and XTEN®2
polypeptidesHigh sustained levels of factor VIII activity with once-weekly dosing with BIVV001
1. In September 2019, Sobi exercised early opt-in for the development and commercialisation of BIVV001, an investigational factor VIII therapy with the potential to provide extended protection from bleeds with once-weekly dosing for people with haemophilia A. Sobi and Sanofi also collaborate on the development and commercialisation of Alprolix and Elocta/ELOCTATE. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory and has manufacturing responsibility for Elocta/ELOCTATE and Alprolix. 2. XTEN is a registered trademark of Amunix Pharmaceuticals, Inc.
BIVV001 is developed and, if approved, will be commercialised in collaboration with Sanofi
Improving patient QoL through once-weekly dosingExtending half-life by factor of 3-4
130%
40%
10%
Factors
NFTs
1 3 7
Fact
or
VIII
leve
ls
Illustrative
Overcoming current limitations in haemophilia treatments
BIVV001 used as monotherapy, whereas NFT require factor to treat breakthrough bleeds
BIVV001 has the potential to achieve more normal factor VIII activity allowing for active lives, whereas NFTs do not
2. SEL-212 gives us a substantial opportunity in immunology
SEL-212’s unique value proposition
Reduced immunogenicity
Monthly dosing
Greater reduction in SUA levels
Sustained efficacy
Better control in patients with tophi
Patient population US (2030)
Diagnosed prevalent gout patients
Refractory gout patients
Chronic refractory gout patients
22
~6.7M
~200K
~190K
~16-20KEligible patients for SEL-212
Source: Sobi
2. The partnership with Apellis is a great example of how we have expanded our late-stage pipeline with multiple opportunities
C3 has a central role in the complement cascade
PNH
CAD
ALS
IC-MPGN/C3G
HSCT-TMA
~7,000-9,000
~9,000-11,000
~8,000-10,000
~6,000-8,000
~200,000
Indications Patient opportunity ex-US (Sobi territory)
23
pegcetacoplan
Source: Sobi
2. Estimated peak sales of our portfolioEstimated peak sales1, SEK B
pegceta-coplan
SEL-2123
pHLHsHLHGraft failureGvHD
ITPCLD
HSCT-TMAALSCADPNHIC-MPGN & C3G
Chronic refractory gout
Haemophilia A+B RSV prevention
BIVV001 is developed and, if approved, will be commercialised in collaboration with Sanofi
MEDI88974
Indications
24
5
7
4-5
8-10
5-12
4-5 3-5 3-4 ~3
RACAPSStill’sNOMID
(incl. royalties)
BIVV0012
1. Peak sales relate to revenue potential from Sobi territories by product 2. BIVV001 is currently under clinical investigation and the safety and efficacy have not been evaluated by any regulatory authority 3. US and Europe 4. Sobi has only financial rights, peak sales based on analyst report Source: Sobi
2. Capturing substantial value from our late-stage pipeline
25
emapalumab, GvHD
emapalumab, pHLH & sHLH
SEL-2123, chronic refractory gout
nirsevimab5, RSV prevention
BIVV0014, HA
pegcetacoplan2, PNH
pegcetacoplan2, CAD
pegcetacoplan2, HSCT-TMA
pegcetacoplan2, IC-MPGN / C3G
avatrombopag, CLD
avatrombopag, ITPCumulated number of planned launches in key geographiesx
5
Launches in key geographies by 2025
32
Key geographies1
4
11
20232022 2024 20252021
19
27
32
1. US, Europe, China, Japan, Russia 2. In collaboration with Apellis 3. Strategic licensing agreement with Selecta 4. In collaboration with Sanofi 5. Financial interest only, in collaboration with Astra Zeneca
Examples of indications (illustrative)
Graph illustrative
3. We are building the next geographic growth platforms
Sobi extended markets
Sobi core markets
Kineret, Orfadin,Alprolix, and Eloctasuccessfully launched
Middle East
Sobi currently has sales of SEK~800M outsidecore markets of Europe and US
Russia
Management team successfully ramped up
Japan
Chinaemapalumaband nitisinonesubmission inprocess
Orfadin launched
Elocta approved
Fully fledged organisation set up
26
Sobi future markets
AustraliaPlanned launchesfor avatrombopag (ITP), emapalumab andpegcetacoplan
In summary: we are now transitioning Sobi from a regional to a global leader in rare disease
27
Lead in Haematology
Grow Immunology
Go global
Capture the value of our
pipeline
Ravi Rao
Head of R&D and CMO
Innovation management
at Sobi
28
Building on Sobi’s rare strength so we can transform patients’ lives
29
Innovative and differentiated medicines• Medicines with novel mechanisms of action: first in class or best in disease• Enabling a step change in therapy for unmet medical need
Leadership in medicines development• Multiple indications and integrated life cycle management• Use of digital health, companion diagnostics and genetics
At the intersection of haematology and immunology• Lead haematology, build immunology and reap synergies between the two• Understanding the needs of patients with rare disease across the world
We currently have 6 products in development, with 12 programs1
301. Not all programs have been started 2. In collaboration with Apellis 3. Strategic licensing agreement with Selecta 4. Financial interest only, in collaboration with Astra Zeneca 5. BIVV001 is developed and, if approved, will be commercialised in collaboration with Sanofi
Phase 2 Phase 3 in Registration
pegcetacoplan2
IC-MPGN and C3G
Gamifant / emapalumab
GvHD
Kineret / anakinra
Deficiency of IL-1 receptor antagonist (DIRA) (US)
SEL-212 / pegadricase3
Chronic refractory gout
pegcetacoplan2
ALS
pegcetacoplan2
CAD
Gamifant / emapalumab
Graft failure (GF)
MEDI8897 / nirsevimab4
RSV Prevention
Doptelet / avatrombopag
Chronic immune thrombo-cytopenia ITP EU
pegcetacoplan 2nd line2
Paroxysmal nocturnal hemoglobinuria and other
BIVV001 / rFVIIIFc-VWF-XTEN25
Haemophilia A
pegcetacoplan2
HSCT-TMA
Gamifant / emapalumab
Secondary HLH rheumatology
Gamifant / emapalumab
Secondary HLH malignancy
Gamifant / emapalumab
Primary HLH RoW
pegcetacoplan 1st line2
Paroxysmal nocturnal hemoglobinuria and other
Novel combination for the treatment of
chronic refractory gout
SEL-212
Single-injection prophylaxis for
all infants in their first RSV
season
MEDI8897
First-in-class treatment for IFNγ-driven
diseases
First-in-class treatment for C3 driven diseases
pegcet-acoplan
Bioengineered to allow for
more normal factor levels with weekly
dosage
BIVV001
Convenient oral thrombopoietin
Receptor Agonist
Six innovative and differentiated medicines to adress patient need across the spectrum of haematology and immunology
31
Lead in haematology Grow in immunologyOverlap between haematologyand immunology
BIVV001 is developed and, if approved, will be commercialised in collaboration with SanofiMEDI8897 is being developed by AZ and commercialized by Sanofi. Sobi has an option to the rights to 50% of the profits and losses of the product in the US
BIVV001 is designed for more normal FVIII levels in HA
pegceta-coplan
SEL 212
MEDI 8897
BIVV001
Designed profile
First product with once weekly dosing and factor level in normal range of FVIII for a sustained period in the week
High levels of physical activity possible with low risk for bleed (e.g. basketball, rugby2) when factor level in normal range
No additional product needed: single administration for prophylaxis through to surgery
Mode of Action
• Built on Fc fusion technology with added
• von Willebrand factor domains
• XTEN1 polypeptides
• To result in high and sustained factor levels in circulation
321. XTEN is a registered trademark of Amunix Pharmaceuticals, Inc. 2. Broderick CR, Herbert RD, Latimer J, et al. Association Between Physical Activity and Risk of Bleeding in Children With Hemophilia.
JAMA. 2012;308(14):1452–1459. doi:10.1001/jama.2012.12727
BIVV001 is developed and, if approved, will be commercialised in collaboration with Sanofi. BIVV001 is currently under clinical investigation and the safety and efficacy have not been evaluated by any regulatory authority.
BIVV001 has the potential to normalise FVIII levels
Disease Background:
• Haemophilia A is a genetic deficiency in clotting factor VIII
• Results in extensive bleeds, joint damage, low QoL
Fact
or
VII
I le
vel,
%
33
50 IU/kg rFVIII (n=28)1 50 IU/kg BIVV001 (n=9)2,3
5
40
0 1 3
100
70
Normal5
130
40
emicizumab4
1. Mahlangu et al. Blood 2014 2. Lissitchkov T et al. Haemophilia 2020;26(S2):55 3. Lissitchkov T et al. Blood 2019;134(S1):625; 4. Equivalent FVIII level, based on Lenting P et al, ISTH 2019, Lenting P et al, Blood Adv. 2020 5. A. Srivastava, E. Santagostino, et al., WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020;26(Suppl 6):1–158
Differentiation:
• BIVV001 has the potential to enable morenormal factor activity levels for the majority of the week
• Short-half life factor therapies do not deliver high sustained factor activity levels
• With emicizumab FVIII normalization is not possible
• Non-factor therapies, e.g., emicizumab, show no evidence of joint protection
Normalisation of factor VIII levels
Days after injection
70
130
BIVV001 is developed and, if approved, will be commercialised in collaboration with Sanofi. BIVV001 is currently under clinical investigation and the safety and efficacy have not been evaluated by any regulatory authority.
Study timeline Key design aspects
Phase 3 in PaediatricN= 65<12 year old
Arms: Multi-centre, open-label 1-arm; previously treated patients with severe haemophilia A
Primary endpoint: Inhibitor development (time frame: baseline to 52 weeks)
2019 2021 20232022
Study Start
Primary Completion
2020
Phase 3 in adults & adolescentsN= 150≥12 year old
Arms: Multi-centre, open-label, non-randomized 2-arm (prophylaxis and on-demand); previously treated patients with severe haemophilia A
Primary endpoint: Annualized bleeding rate (ABR) in prophylaxis treatment arm (time frame: baseline to 52 weeks)
2019 2021 20232022
Study Start Primary Completion
2020
Two major phase 3 studies underway - BIVV001 expected to be submitted in US in 2022
341. Only territories relevant to Sobi displayed
Expected launch1
2023 Haemophilia A
Doptelet could be a best-in-class treatment for CLD and ITP
Profile• Once daily oral dosing
• No dietary restrictions - convenient administration with food/regular meals
• No hepatotoxicity
Mode of Action• Second-generation small-molecule
thrombopoietin receptor agonist (TPO-RA) stimulating platelet production
• Stimulates proliferation and differentiation of megakaryocytes from bone marrow progenitor cells resulting in increased production of platelets
pegceta-coplan
MEDI 8897
SEL-212
BIVV001
35
Doptelet can increase platelet numbers to significantly reduce need for platelet transfusions
Disease background:
• Low levels of blood platelets can cause spontaneous bleeding
• Thrombocytopenia is caused by, e.g.:
- Decreased production (e.g., chronic liver disease (CLD))
- Increased destruction (e.g., immune thrombo-cytopenic purpura)
36
Current TPO-RA treatments:
• eltrombopag, romiplostim
Differentiation:
• Patient convenience: Doptelet as the only once daily oral TPO-RA approved for treatment of ITP without dietary restrictions1
• No hepatotoxicity
Median platelet counts during trial2
Titration Concomitant ITP Med Reduction MaintenanceavatrombopagPlacebo
Randomization Day
Pla
tele
t C
ou
nt
(x 1
09/l
)
Study Visit Time (in weeks related to Randomization Day)
32 30 32 32 32 32 30 30 30 28 25 28 25 24 22 25 22 23 23 23 22avatrombopag (n)
Placebo (n) 17 17 17 17 16 16 14 7 4 3 3 3 3 2 2 2 2 1 1 1 1
1. Approved for ITP in the US and awaiting approval in the EU See prescribing information in the applicable territory for the approved indication 2. Jurczak, et al. Br J Haematol. 2018;183(3):479-490
We have finished the majority of the development with focus on ongoing lifecycle studies
Study timeline Key design aspects
Early use study adults ITPN= 75
Arms: Pbo – 2:1 randomization with 6-month duration of treatment
Primary endpoint: Continuous weeks of response (PC>50)
Q2/2021
Study Start
Q4/2023
Primary Completion
2019 2022 2024202320212020
Phase 3 in paediatric ITP N= 72 <18 year old
Arms: Randomized 3:1 to receive either avatrombopag or placebo (orally once daily for 12 weeks)
Primary endpoint: Proportion of subjects achieving at least 6 out of 8 weekly platelet counts ≥50×109/L during the last 8 weeks of 12-week treatment, in the absence of rescue medication
12/2020
Study Start
05/2023
Primary Completion
2019 2022 2024202320212020
Expected launch
2021 ITP
37
SEL-212 is a novel combination product candidate to improve treatment of chronic refractory gout
ImmTOR™1 has the potential to enable sustained therapeutic activity of biologic therapies and unlock their potential
SEL-212: ImmTOR™ co-administered with pegadricase
Rapamycin
ImmTOR™ PLA+PLA-PEG
pegadricase
Uricase
PEG
Mode of Action• ImmTOR™ co-administered with
pegylated uricase to reduce serum uric acid (SuA) and inhibit antibody development
Designed profile• Once-monthly infusion
• Reduced immunogenicity
• Sustained efficacy
pegceta-coplan
SEL-212
MEDI 8897
BIVV001
38
Biologic alone
1. ImmTOR™ is a registered trademark by Selecta Biosciences, Inc.
Phase 2 results show potential for greater reduction of SUA COMPARE trial results (SEL-212 vs pegloticase)1
Disease Background:
• Chronic gout: serum uric acid levels remain elevated and flares continue despite standard treatment
• Painful inflammatory arthritis: mono- or polyarticular
• Presence of subcutaneous tophi
Current Treatments:
• pegloticase; efficacy rates of ~42%;
- ~50% of patients can tolerate full treatment course (6m)
Patients with <6 mg/dl SUA for at least 80% of the evaluation time
Month 3
Month 3 and 6 combined2
Primary study endpoint
pegloticaseSEL-212 p-value
70%
51%
PP1
0.019* exploratory
PP3
0.056
59%
46%
39
Differentiation:
• Less frequent dosing (monthly vs every other week)
• Statistically significantly greater overall reduction in mean serum uric acid (SUA) levels in SEL-212 versus pegloticase
• Higher efficacy rates in patients with tophi versus pegloticase
• No need for oral immunosuppression with MMF or MTX
Note: Per FDA guidance on Statistical Considerations for Clinical Trials During the COVID-19 Public Health Emergency (June 2020), the statistical analysis plan was modified and submitted to FDA prior to database lock to address the potential impact of the COVID-19 pandemic on statistical analysis. This was necessary due to increased protocol deviations in the intention-to-treat (ITT) population observed during the ongoing COVID-19 pandemic. Data are therefore presented per protocol (PP) and ITT1. Press release by Sobi and Selecta Biosciences on topline data of SEL-212 from phase 2 COMPARE study 2. SEL-212 showed a numerically higher response rate on the primary endpoint during months 3 and 6 combined, but did not meet the primary endpoint of statistical superiority 3. Presented per protocol
Patients with tophi have significantly higher responder rates for SEL-212 as compared to pegloticase based on phase 2 dataChronic refractory gout patients Study results for tophi patients1
40
Treatment response rates2
(PP, months 3 and 6 combined) Serum Uric Acid (SUA) percent reduction3
(PP, baseline and months 3, 6)
pegloticaseSEL-212
58%
39%
1. COMPARE study 2. Patients with <6mg/dl SUA for at least 80% of evaluation time 3. Computed as the mean SUA level during treatment period minus baseline SUA level divided by baseline SUA level multiplied by 100
>70% have visible tophi US (2030)
27%
26%
47%
1 visible tophus
>1 visible tophi
Without visible tophi
~192K
~90K
~50K
~52KBaseline Month 3 Month 6
-100
-80
-60
-40
-20
0
20
%
SEL-212
Pegloticase
-61% -57%
-77% -77%
SEM
41
Two phase 3 studies on SEL-212 have been launched this year
Study timeline Key design aspects
Dissolve 1Study 1N= 105
Arms: Randomized 1:1:1 to receive either one of two SEL-212 dose levels or placebo every 28 days for 6 months
Primary endpoint: Serum uric acid control during month 6
Extension: 6 months
Dissolve 2Study 2N= 105
Arms: Randomized 1:1:1 to receive either one of two SEL-212 dose levels or placebo every 28 days for 6 months
Primary endpoint: Serum uric acid control during month 6
2020 2021 20232022
2020 2021 20232022
08/2020
Study Start
03/2022
Primary Completion
10/2020
Study Start
05/2022
Primary Completion
41
Expected launch
2023Chronic Refractory Gout
About Synagis and MEDI8897
pegceta-coplan
MEDI 8897
BIVV001
• The 2018 acquisition from AstraZeneca included rights to Synagis® (palivizumab) in the US as well as rights to participate in 50 per cent of the future earnings of the candidate drug MEDI8897 in the US
• Synagis is a medicine for the prevention of serious lower respiratory tract infections (LRTI) caused by respiratory syncytial virus (RSV) in high-risk infants and is the only approved preventative medicine for the condition
• MEDI8897 is a follow-on candidate to Synagis and a monoclonal antibody (mAb) being investigated for the prevention of LRTI caused by RSV in a broad infant population
• MEDI8897 derisks future revenue expectations for Synagis
42
SEL-212
MEDI 8897
MEDI8897 is being developed by AZ and commercialised by Sanofi. Sobi has an option to the rights to 50% of the profits and losses of the product in the US
Profile • Approved for primary HLH in US in Q4 20182
• Three further indications in development: secondary HLH, Graft Failure and GvHD
Gamifant is the only treatment to target and neutraliseIFNγ – a major proinflammatory cytokine
Mode of action• Monoclonal antibody to neutralise
interferon gamma (IFNγ)
• Aberrant IFNγ expression is associated with a number of autoinflammatory and autoimmune diseases such as HLH1 – a severe, sometimes life-threatening systemic inflammatory syndrome
1. Hemophagocytic lymphohistiocytosis. 2. Approved for the treatment of adult and pediatric patients with primary HLH with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy, in US. See Gamifantprescribing information, FDA.
Sources: Jordan MB, et aI.: How I treat hemophagocytic lymphohistiocytosis. Blood. 2011; Price B et aI. Haemphagocytic lymphohistiocytosis: a fulminant syndrome associate with multiorgan failure and high mortality that frequently masquerades as sepsis and shock. S Afr Med J. 2014
pegceta-coplan
MEDI 8897
SEL-212
BIVV001
43
IL-1β
IL-18
ApoptosisInflammasome activation
NLRC4
Granzyme B
Perforin PRF1
Priming MUNC13-4
Fusion STXBP2STX11
DockingRAB27a
PolarizationAP3BP1
Vesicular protein Sorting
LYST
IFNγ
IL-1R
IL-18R
IL-33/ST2
Antigen presenting cell Cytotoxic Lymphocyte
Disease progression
pHLH is a life-threatening disease caused by genetic mutations
Disease Background1:
• Caused by multiple genetic mutations,
• ~55% described, ~45% unknown
• Higher prevalence among children
• Mostly triggered by infection
Current Treatments and differentiation:
• Gamifant is currently the only FDA approved therapy
Differentiation:
• Seeking approval in other countries (ex-EU)
• Further exploration of genetic databases to further inform patient care
44
Overall response in previously treated patients was significantly higher than the prespecified null hypothesis (p=0.02)
At last observation, 20/27 (74%) patients were alive with an estimated probability of survival of 73.4% (95% CI 52.2−86.4) at 12 months
0
80
20
60
40
Complete Response
Percent of patients
Overall Response
Partial Response
HLH Improvement
63.0
26.0 30.0
7.0
NI-0501-04 Study: response at wk 8 (end of treatment)2
Patients with inadequate response to therapy (N= 27)
NI-0501-05 Study: survival at 12m2
0.8
0
0.4
0.2
0.6
1.0
110 171 202 3 4 5 6 87 9 10 1612 13 14 15 18 19
1. Jordan et al Blood 2011; 118 (15): 4041 2. Locatelli et al N Engl J Med 2020; 382 (19): 1811
We are expanding emapalumab in HLH and beyond into diseases of high unmet need
45
HLH
Move to the next frontier:GF and GvHD post-HSCT
Precision medicine: Development in conjunction with a companion diagnostic
Maximize pHLH
Expand geographically
Continue to drive disease awareness and accelerategenetic understanding
Expand into sHLH
Development of secondary HLH driven by cytokine genetic and clinical data:
Include rheumatologic HLH, malignant HLH and infectious HLH
HSCT
Expected launch
2021-2022 pHLH
2023 sHLH
2024 GvHD
46
Many cases of HLH lie on a spectrum between sHLH and pHLH
We are gaining a deeper understanding of the impact of unknown genetic mutations and infectious triggers in the pathogenesis of HLH
Known Genetic Mutations (55%)
Perforin, Inflammasome, Immune deficiency
“Primary” HLH “Secondary” HLH
Genetic mutationInfection, Rheumatologic, Malignancy
Unknown Genetic Mutations (45%)
FeverSplenomegaly
HypofibrinogenimiaHypertriglyceridemia
CytopeniasHyperferritinemia
Low NK cell activityElevated sCD25/sILRa
Common phenotype
Infection: e.g., EBV, COVID-19, CMV, Iatrogenic, otherTriggers
1. Source: Jordan et al Pediatr Blood Cancer 2019; 66 (11): e27929
Emapalumab could be a break-through treatment for rheumatologic HLH
Disease Background:
• Severe, potentially fatal condition associated with excessive activation of macrophages and T cells leading to an overwhelming inflammatory reaction1
Current Treatments:
• No approved treatment to prevent or arrest rheumatological HLH (MAS in sJIA2) and AOSD
1. Zhang et aI. Arthritis Rheum. 2008 2. Macrophage Activation Syndrome in Systemic Juvenile Idiopathic Arthritis
Differentiation:
• First IFN-targeted therapy for rheumatological HLH
Study NI-0501-06 MAS in SJIA/AOSD
• Gamifant started on day 0:
- 14/14 patients had a clinical response
- Improvement in multiple lab parameters
47
ALT (IU/L)
Time(day) Time(day)
Ferritin (UG/L)
10
50
200
1000
5000
0 5 10 15 20 25 300 5 10 15 20 25 30
1e+01
1e+03
1e+05
Time(day)
AST (IU/L)
5
20
100
500
5000
0 5 10 15 20 25 30
Time(day)
CXCL9 (pg/ml)
0 5 10 15 20 25 30
5e+01
5e+02
5e+03
5e+04
We are carrying out an extensive development plan for sHLH
4848
Timeline
rHLH
PilotN= 14
PivotalN= 30-40
mHLH
PilotN= 10-15
iHLH
Key design aspects
Arms: Single-arm, open label
Endpoints: MAS remission, duration of response
Arms: Single-arm, open label, observational run-in phase
Endpoints: MAS remission, duration of response
Arms: Single-arm, open label, adaptive
Endpoints: ORR, survival, duration of response
Genetic data mining/phenotype identification will guide design and patient selection criteria
2019 2020 20222021 2023 2024
Genetic &cytokine data
iHLH
2019 2020 20222021 2023 2024
2019 2020 20222021 2023 2024
Data for US Data for ROW
Decision point: Based on results of pilot
FDA meeting
Development of emapalumab for graft failure to start in Q1 2021
Disease Background:
• Primary GF: lack of engraftment of donor cells following Hematopoietic stem cell transplantation (HSCT); high mortality
• Secondary GF: loss of graft after initial engraftment
Current Treatments:
• Repeat graft
Source: Merli et al., 2019. Haematologica. Volume 104(11):2314-2323
Differentiation:
• First IFN-targeted therapy for immune-related graft failure
49
D0: HSCT
D100D30 to
D421 2 3
1.Primary GF (pGF)Transplanted cells
never engraft
2. Secondary GF (sGF)Loss of graft after initial engraftment
3. Acute Graft versus Host Disease (aGvHD)Transplanted cells have immune reaction
against the patient
Three possible complications following HSCT could be treated with emapalumab
Collaboration with bioMérieux to develop a Companion Diagnostic
Research collaboration for a CXCL9 companion diagnostic assay for Gamifant for GvHD
Note: CoDx = companion diagnostics; HSCT = Hematopoietic stem cell transplantation
Fast turnaround and hands off
Could predict graft failure following HSCT as well as future indications
50
Increased serum levels of IFNγ and CXCL9 are predictors of graft failure
• As early as day 3 post-transplantation, patients developing GF had elevated serum CXCL9
• Serum CXCL9 appears to be an early biomarker for the risk of graft failure
CXCL9 (pg/mL)2
Time from HSCT (days)
100 000
10 000
1 000
100
103 307 10 14
ControlsGF
VIDAS™1 CXCL9 for the prevention of graft failure post HSCTreceived breakthrough device designation by FDA inMay 2020
1. Vidas™ is a registered trademark of bioMerieux 2. Merli et al., 2019. Haematologica
Enabling development of emapa-lumab in GvHD to start in 2021
Disease Background:
• GvHD is a potentially serious complication of allogeneic HSCT
• Donor T cells attack the host which leads to multi-organ damage and can be life-threatening
Current Treatments:
• Ruxolitinib currently approved
Source: Merli et al., 2019. Haematologica. Volume 104(11):2314-2323, McCurdy et al., 2020 51
Differentiation:
• First IFN-targeted therapy for GvHD resistant to SOC
CXCL9 levels at d28 post transplant are predictiveof 1 year cumulative incidence of aGvHD
Development program for GvHD to start in 2021 with expected launch in 2024
5252
Graft failure (GF)
Observational study 13 (N= 200-300)
PoC study 12 (N= up to 36)
Pivotal study GF
GvHD
VIDAS
GvHD NH cohort
Pivotal study GvHD
Key design aspects
Design: Allogeneic HSCT patients from HSCT to day 100 to support CoDx development, CXCL9 cut off for GF and GvHD pivotal studies
Endpoints: IFNG and CXCL9 levels
RCT l in patients at high risk of primary and/or secondary GF to demonstrate reduction of GF rate
Design: Single-arm, open label, adaptive to confirm dose & efficacy in high-risk patients
Endpoints: PKPD, patient proportion with GF
Registration study in 2nd and/or 3rd line in steroid-resistant and/or ruxo-resistant patients
Enabling activities to inform study design and targeted population in aGvHD
Deliver investigational use only device ready for clinical validation prior to phase 3
Companion Diagnostic
Timeline
2020 20272021 20232022 2024 20262025
2020 20272021 20232022 2024 20262025
2020 20272021 20232022 2024 20262025
Anakinra & emapalumab have the potential to address key pathways in COVID-19 CSS1
53
Immuno-101 POC study of anakinra or emapalumab vs. SOC has been stopped
Ongoing clinical trial activity with anakinra:
• 18+ ongoing or planned RCT studies of anakinra in moderate-severe disease
• 10 studies supported by Sobi across US and EU; 1,000/~2,500 patients recruited
1. Fajgenbaum et al N Engl J Med 2020; 383: 2255 2. Soluble urokinase-type plasminogen activator receptor
Pegcetacoplan is a targeted C3 therapy – it is therefore modulating a key component of the complement system
Mode of Action
• Investigational, targeted C3 therapy designed to regulate excessive complement activation
• Uncontrolled complement activation is involved in the pathology of a broad range of disorders including various autoimmune and immune complement driven diseases
Designed profile• Potential to elevate the standard of care in PNH
• Met primary endpoint and safety profile consistent with eculizumab in PEGASUS trial
• Subcutaneous administrationpegceta-coplan
MEDI 8897
SEL-212
BIVV001
54
Initiation
Amplification
Termination
Proximal complement
Classical pathway
Lectin pathway
Alternativepathway
Terminal complement
C5 activation
C5 inflammation
HaemolysisOpsonisation of red blood cells
C3 activationPegcetacoplan
On-market C5 inhibitors
The complement system underlies many diseases; in addition to PNH, 3 others are part of the initial focus for pegcetacoplan
Source: Modified based on Zelek et al., Compendium of current complement therapeutics, Molecular Immunology 114 (2019) 341–352 55
SURGERY-RELATEDI/R injury, AKI, Organ transplantation
ARTERIES/VESSELSAtherosclerosis
GUTCrohn's Disease
LUNGSAsthma, COPD, ARDS
MOUTHPeriodontitis
PNS/CNSAD, MS, NMO, gMG, GBS, Parkinson's Disease, Schizophrenia
EYEAMD, Uveitis, Glaucoma
HEARTMyocardial infarction
KIDNEYaHUS, LN, IgAN, MN
JOINTS/SKINRA, OA, BP, Psoriasis, HS, Burns
TREATMENT-RELATEDHaemodialysis
SIRS, Sepsis, Trauma, SLE, HAE, Cancer
CIRCULATIONPNH, TMAs, AAV
Pegcetacoplanfocus indications
IC-MPGN/ C3G
PNH, CAD, HSCT-TMA
ALS
Nephrology
Neurology
Haematology
Paroxysmal Nocturnal Haemoglobinuria (PNH): Superior improvement in Hb compared to eculizumab1
Disease Background (PNH):
• Acquired, rare, chronic, life-threatening blood disorder associated with abnormally low hemo-globin levels and transfusion dependence due to hemolysis
• Symptoms include severe fatigue, abdominal pain, difficulty breathing, and hemoglobinuria
Current Treatments:
• C5 inhibitors
PEGASUS Study vs eculizumab: Week 16
Change from baseline hemoglobin in patients with a suboptimal response to eculizumab
56
Differentiation:
• Superiority on improving Hb vs eculizumab due to effects on intra- and extra-vascular haemolysis
• Substantial improvement in other haemato-logical and clinical parameters vs eculizumab
1. Based on PEGASUS-study: 71% of pegcetacoplan-treated patients achieved LDH normalization vs. 15% of eculizumab-treated patients. LS Mean (+/-SE) plot of change from baseline in hemoglobin using MMRM model over time – randomized controlled period (ITT set)
Δ 3.8 g/dL at
week 16,
p<0.0001
pegcetacoplan
eculizumab
Highly targeted treatment in Cold Agglutinin Disease (CAD)
Disease Background:
• Chronic and severe red blood disorder driven by extravascular hemolysis (IgM)
• Symptoms include anemia, transfusion requirements, and increased risk of thrombotic events like stroke or heart attack
Current Treatments:
• No therapies approved or indicated to treat CAD
Source: 1. Catenion using physician and literature consensus.. 2. Fattizzo B, et al. European Hematology Association. June 13-16, 2019. 57
Differentiation:
• Expected to be first targeted C3 treatment for CAD
Interim Results: PLAUDIT Study, a phase 2 study n=24
12
7
14
8
9
11
10
13
CAD hemoglobin, g/dL
Rapid, sustained, and durable increase in hemoglobin in response to pegcetacoplan
LLN
714 281 56 84 112 140 168
Day
13
1313
11 1211
11 910
Normal range 12-17.5 g/dL
n=
One of the first targeted therapies in HSCT-associated Thrombotic Microangiopathy (TMA)
Disease Background:
• HSCT-TMA is a rare inflammatory and thrombotic condition characterized by hemolytic anemia, thrombocytopenia, and evidence of multiorgan damage, particularly renal dysfunction1
• C3 is believed to play a critical role in TMA based on proinflammatory and procoagulant properties of C3a and C3b2
Current Treatments:
• No approved therapies
1. Dvorak C, et al. Frontiers in Pediatrics. 2019, Vol 7, article 133 2. Noris M, et al. Nature Reviews Nephrology. 2012, 8: 622–633 58
Differentiation:
• First targeted C3 treatment for HSCT-TMA
Elevated C3b levels in patients developing TMA post-HSCT
Qi et al. 2017
166+/-98
Before After
455+/-224
C3b (ng/mL)
Mean Median
Before
138(102-188)
After
394(300-519)
First line treatment in IC-MPGNand C3 Glomerulopathy1
Disease Background:
• Rare kidney diseases caused by excessive complement activation that can lead to kidney failure
• C3G is associated with a need for recurrent transplant in as many as 85%
• Classical and alternative pathways implicated in IC-MPGN
Current Treatments:
• No approved therapies
DISCOVERY Study: Week 48*
*In five C3G patients; three patients were excluded from the analysis due to self-reported non-compliance or study drug interruption
24-hour uPCR, mg/mg
BaselineMean (SE)
3.48 (0.82)
Week 48Mean (SE)
0.93 (0.27)
Difference
(73.3%)
59
Differentiation:
• First targeted C3 treatment for C3G
1. Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN) and C3 Glomerulopathy (C3G)
Potentially registrational phase 2 study design laid out for ALS1
Disease Background:
• Neurodegenerative disease that results in progressive muscle weakness and paralysis due to the death of nerve cells in the brain and spinal cord
• High levels of C3 throughout motor system of patients may contribute to neuroinflammation and death of motor neurons
Current Treatments:
• No approved therapies have been shown to stop or reverse disease progression
Source: Arthur K et al., Nat Commun, 2016, Vol 7, article 12408
Potentially Registrational Phase 2 Study Design
1 year randomized, placebo-controlled
1 year open-label
CAFS primary endpointreadout
Primary endpoint: Combined Assessment of Function and Survival at Week 52
Secondary endpoints: Measures of lung function, muscle strength, and quality of life
Design: Double blinded, randomized 2:1
Sample size: ~200 patients with sporadic ALS
Duration: 2 years
60
Differentiation:
• First targeted C3 treatment for ALS
1. Amyotrophic Lateral Sclerosis (ALS)
61
Our clinical development program is on the way to launch pegcetacoplan in 5 indications by 2024
Expected launch
2021 PNH
Category Disease
Nephrology IC-MPGN / C3G
Haematology PNH
CAD
HSCT-TMA
Phase 2 Phase 3
Neurology ALS
Initiate phase 3 study in 2021
Initiate phase 2 study in 2021
Initiate phase 3 study in 2021
Sobi leads development
Capturing substantial value from our late-stage pipeline
62
emapalumab, GvHD
emapalumab, pHLH & sHLH
SEL-2123, chronic refractory gout
nirsevimab5, RSV prevention
BIVV0014, HA
pegcetacoplan2, PNH
pegcetacoplan2, CAD
pegcetacoplan2, HSCT-TMA
pegcetacoplan2, IC-MPGN / C3G
avatrombopag, CLD
avatrombopag, ITPCumulated number of planned launches in key geographiesx
5
Launches in key geographies by 2025
32
Key geographies1
4
11
20232022 2024 20252021
19
27
32
1. US, Europe, China, Japan, Russia 2. In collaboration with Apellis 3. Strategic licensing agreement with Selecta 4. In collaboration with Sanofi 5. Financial interest only, in collaboration with Astra Zeneca
Examples of indications (illustrative)
Graph illustrative
4 Centers of excellence with ~250 team members• deepening experience of haematology and immunology
Basel
DurhamBoston
Building our rare strength in R&D
A portfolio across a broad range of rare haematologic and immunologic diseases
Innovative and differentiated medicines
Deep experience with collaboration and partnership
Asset development in multiple indications and utilising leading enabling technology
Sobi leadership in areas of expertise:
• emapalumab in sHLH, GF, GVHD (HSCT)
• pegcetocoplan in CAD and TMA (HSCT)
• anakinra in COVID19
Stockholm
Norbert OppitzInternationalisation
Strategy
64
The rare disease market represents compelling opportunities outside Europe and US
78
118
33
51
35
55
2019
223
2024
145
International USEurope
Strong double-digit market growth for rare diseases outside current Sobi core territories
>250 million people affected by rare diseases excluding US/EU, thereof 60% children
Sobi committed to reach 80% of affected patients with Sobi rare disease products
Key facts
Source: Profound
Rare disease market, USD B
X CAGR
+9%
85% are serious /life-threatening
~85%
Only approximately 500 approved drugs to date
500
7,000+More than 7,000 designated rare diseases exist
65
Our Mission 2025 goal is to go global and make our products available to twice as many patients as today
Mission 20252017-2020
Becoming a regional leader in rare disease
Global leader in rare disease
Establish US as a second pillar next to Europe
Started to further internationalise, e.g. Middle East
Presence established in key markets China, Japan, Russia and Australia
Partnerships to serve underserved regions
66
We have already expanded our market presence outside Europe and the US
Sobi extended markets Sobi future marketsSobi core markets
Alprolix and Elocta successfully launched
Middle EastChinaemapalumab andnitisinonesubmission in process
Orfadin launched
Russia
Elocta approved
Leadership team identified andorganisationalramp-up ongoing
Japan
Fully fledged organisation set up
Sobi has already established presence in Middle East, Russia and China
Current sales of SEK ~800M outside its coremarkets (Europe and US)
Second wave now focuses on Japan, Australia, South-East Asia and Latin America
67
AustraliaPlanned launches for avatrombopag (ITP), emapalumab andpegcetacoplan
International will ramp up value with a high cadence of launches
681. Regions outside NA and Europe 2. In collaboration with Apellis 3. Strategic licensing agreement with Selecta
202420232022 20252021 SEL-2123, chronic refractory gout
emapalumab, sHLH
avatrombopag, CLD
pegcetacoplan2, PNH
avatrombopag, ITP
pegcetacoplan2, C3G
pegcetacoplan2, CAD
emapalumab, GvHD
emapalumab, pHLH
nitisinone, HT-1
eloctate, HA
Potential launchesin international
regions1 by 2025
18+
Examples of indications (illustrative)
Graph illustrative
Example: China – we are fast-tracking to enable patients to access emapalumab and nitisinone in 2022
410
660
2020 2030
pHLH
2020 2030
sHLH
4.500
5.600
530
720
20302020
<6y130
360
>6y
20302020
emapalumab progress nitisinone progress
+
HLH patients in China
Mar 20 Jun 20 Dec 20 Q3-4 21
China adboardon HLH
CDE meeting on emapa-lumab
NDA submission emapa-lumab
Potential approval (priority review)
Jun 20 Jun 20 Dec 20 Q3 21
China adboardon HAT-1
CDE meeting on nitisinone
NDA submission nitisinone
Potential approval (priority review)
HT-1 patients in China
Source: Sobi
Legal entity established
GM with 20+ years of experience
Medical support established
Supply chain designed
Regulatoryexpertise onboarded
69
Launch of anakinra in CAPS expected 2023
Example: Japan – en route to introduce emapalumab, anakinra, avatrombopag and pegcetacoplanPatient pools by indication
High prevalence of HLH-KD (Kawasaki) and HLH-EBV (Epstein-Barr) – filing under evaluation
Total 870
70
1,024
pHLH
800sHLH
82
942
ITPTPO-RA
CLD1
9,000
14,000Total 17,297
5,000
12,304
4,9932
5,021
2,500
7,005
sJIA
Total
AOSD 4,500
100CAPS
7,910
2,789
952
20302019
Well-established markets already
Current TPO-RA market at about USD 300M
Build experienced leadership team and scaled-up organisation
70
185
185
Total
PNH
228
228
emapalumab anakinra avatrombopag pegcetacoplan
2025
2022
2023
2022
2024 2023
1. CLD patients receiving surgery 2. Decrease in line with population expected
Source: Scientific research, journals, government publications, patient surveys, practice guidelines, publications from associations, expert interviews, claims data
Launch year
By 2025, International is expected to contribute 15% of Sobi's revenue
>4
2020
Internationalrevenue
2025
0.8
+40% p.a.
Sobi extended markets Sobi future marketsSobi core markets Revenuein SEK B
Share of total revenue
~5% ~15%
71Source: Sobi
Henrik Stenqvist
CFOFinancial update
72
Operational performance in 2016 – 2020 has generated strong revenue, EBITA and cash flow
2016 2017 2018 2019 2020E
9.1
5.26.5
14.215.0-15.5
+10
Specialty Care Immunology Haematology
Total Revenue, SEK B
32% 39% 43%
x
~39%
Adj. EBITA
2020E based on mid-point guidance provided by Sobi in Q3 2020 report
Declining revenues due to partner portfolio terminations
Orfadin decline due to generics
Specialty Care
Achieved continued growth in Haemophilia
Realised successful launch of Doptelet
Haematology
Maintained stable ~40% margins over the last 3 years
EBITA
Impact from Synagis acquisition and successful Gamifant launch
Realised growth in Kineret
Immunology
30%
73
Continued strong cash generation
Cash flow
Sobi is in a new stage as a company
74
Mission 2025
Global leader in rare disease
Competitive situation within Haemophilia
We now have a significant pipeline of late-stage assets to be brought to approval
Commercial success will be driven by realizing the potential of launches on a global scale
We will continue to make strategic M&A
74
Bringing assets to approval and realising commercial potential on a global scale
75
R&D spend – pipeline of late-stage assets to be brought to approval
• Moving forward in 2021-2022, will increase to 13-15% of sales driven by investments in:
• SEL-212
• Pegcetacoplan across several indications
• Emapalumab indication expansion into GF and sHLH
• Continued investments in Doptelet and Gamifant
• Launch of SEL-212, pegcetacoplan and BIVV-001
• Further investments in infrastructure in international markets
SG&A spend – realising the potential of launches on
a global scale
2023 – 2025:• Continuous potential double-digit revenue growth• Mid-term revenue aspiration of 25B SEK by 2025• EBITA margin acceleration
Growth acceleration from 2023 onwards
15 – 15.5B
2020 2021 20242022 20252023
25B
Revenue CAGR of +10%
Revenue (absolute) EBITA (absolute)
2021 – 2022: • Single-digit revenue growth • Investment into the future related
to R&D, launches and internationalisation
• Pressure on EBITA
76
A continued strong cash flow will support growth through M&A
Continued strong cash generation with seasonality driven by Synagis
Net debt / EBITDA will likely be below 3 in short-term
Ability to lever up to 3-4x range
77
0
10
20
Q3 2019 Q1 2020 Q3 2020Q2 2019Q1 2019 Q2 2020Q4 2019
-2.7B
SEL-212Acqui-sitions
Net debt, SEK B
Focus M+A in strategic areas –derisk where possible
Commercial
30%Regulatory
70%
35%2020 - 2025
65%Beyond 2025Included FDA priority review voucher with high re-
sale value de-risked by multiple potential indications
3 indications, bulk of purchase value from CLD and ITP
Limited upfront payment; future payments contingent on R&D and commercial success
SEL-212
Example actions to de-risk
Financial stake in follow-on MEDI8897
Pegceta-coplan
Limited upfront payment; future payments contingent on R&D and commercial success
Majority of obligations relate to commercial milestones
Majority of obligations estimated due after 2025
78
Guido Oelkers
CEOWrap-up and Q&A
79
Conclusion
Proven track record - since 2016 we have created a strong foundation with regard to revenue, EBITA and cash flow, whilst building the organisation and our pipeline
Next phase requires investment into development, pre-launch and launches of our pipeline to propel mid- to long-term growth
Further M+A will be explored
Our ambition is to reach SEK 25B revenues in 2025
80